19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 899 Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

  
8 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
Inclusion_
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PhaseCountries
1EUCTR2010-020048-36-GB
(EUCTR)
31/01/201117/06/2010A safety, tolerability and preliminary efficacy study of idursulfase-IT in patients with Hunter syndrome associated with learning disabilityA Phase I/II, Randomized, Safety and Ascending Dose Ranging Study of Intrathecal Idursulfase-IT administered in conjunction with intravenous Elaprase in Pediatric Patients with Hunter Syndrome and Cognitive Impairment Treatment of Hunter syndrome and cognitive impairment
MedDRA version: 14.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Idursulfase (I2S)-IT
Product Code: I2S-IT
INN or Proposed INN: NA
Other descriptive name: Idursulfase-IT
Shire HGT, IncNULLNot Recruiting Female: no
Male: yes
20 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
2EUCTR2004-002743-27-IT
(EUCTR)
26/07/200510/08/2005An open-label extension of study TKT024 evaluating long-term safety and clinical outcomes in MPS II patients receiving iduronate-2-sulfatase enzyme replacement therapy.An open-label extension of study TKT024 evaluating long-term safety and clinical outcomes in MPS II patients receiving iduronate-2-sulfatase enzyme replacement therapy. Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 6.1;Level: PT;Classification code 10056889
Product Name: Idursulfase
Product Code: I2S
TKT INCNULLNot RecruitingFemale: no
Male: yes
94United Kingdom;Germany;Spain;Italy;Sweden
3EUCTR2004-002743-27-SE
(EUCTR)
11/05/200517/03/2005An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 6.1;Level: PT;Classification code 10056889
Product Name: Idursulfase (I2S)
Other descriptive name: Idursulfase
Shire Human Genetic Therapies INC.NULLNot RecruitingFemale: no
Male: yes
96United Kingdom;Germany;Spain;Italy;Sweden
4EUCTR2004-002743-27-ES
(EUCTR)
21/04/200504/04/2006An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 6.1;Level: PT;Classification code 10056889
Product Name: Idursulfase (I2S)
Other descriptive name: Idursulfase
TKT Inc.NULLNot RecruitingFemale: no
Male: yes
94Phase 3Spain;Germany;Italy;United Kingdom;Sweden
5EUCTR2004-002743-27-GB
(EUCTR)
31/01/200523/02/2005An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 6.1;Level: PT;Classification code 10056889
Product Name: Idursulfase (I2S)
Other descriptive name: Idursulfase
TKT Inc.NULLNot RecruitingFemale: no
Male: yes
94Phase 3Spain;Germany;Italy;United Kingdom;Sweden
6EUCTR2004-002743-27-DE
(EUCTR)
23/12/200430/11/2004An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement TherapyAn Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 6.1;Level: PT;Classification code 10056889
Product Name: Idursulfase (I2S)
Other descriptive name: Idursulfase
Shire Human Genetic Therapies INC.NULLNot RecruitingFemale: no
Male: yes
96United Kingdom;Germany;Spain;Italy;Sweden
7EUCTR2011-000212-25-Outside-EU/EEA
(EUCTR)
19/02/2014An extension to the safety, tolerability and preliminary efficacy study of Idursulfase-IT in patients with Hunter syndrome associated with learning disabilityAn Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Pediatric Patients with Hunter Syndrome and Cognitive Impairment Treatment of Hunter syndrome and cognitive impairment
MedDRA version: 16.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: Idursulfase(I2S)-IT
Product Code: I2S-IT
INN or Proposed INN: IDURSULFASE
Other descriptive name: idursulfase-IT
Shire HGT IncNULLNAFemale: no
Male: yes
15Canada;United States
8EUCTR2007-006044-22-PL
(EUCTR)
21/04/2008A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not availableA Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not available Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome)
MedDRA version: 10.1;Level: PT;Classification code 10056889;Term:
Trade Name: Elaprase 2mg/ml
Product Name: Idursulfase (I2S)
Product Code: I2S
INN or Proposed INN: Idursulfase
Other descriptive name: Idursulfase
Shire Human Genetic Therapies, Inc. (Shire HGT)NULLNot RecruitingFemale: no
Male: yes
30Phase 4Poland